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Celldex Provides Corporate Update and Reports First Quarter 2019 Results

On May 7, 2019 Celldex Therapeutics, Inc. (NASDAQ:CLDX) reported business and financial highlights for the first quarter ended March 31, 2019 (Press release, Celldex Therapeutics, MAY 7, 2019, View Source [SID1234535819]).

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"Celldex presented positive data across multiple programs at AACR (Free AACR Whitepaper) in April, including from our promising CDX-1140 program," said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. "We have successfully cleared a critical hurdle for CD40 agonists, reaching dose levels with good systemic exposure that are biologically active and well tolerated. Importantly, these dose levels exceed the maximum tolerated dose levels reported with other CD40 agonists, which we believe may support enhanced tissue and tumor penetration. We are also pleased with the results to date in our unique combination of CDX-1140 with CDX-301, where CDX-301 amplifies the numbers of dendritic cells in patients prior to their activation with CDX-1140. To this end, we continue to believe that CDX-1140 can play a very important role in cancer immunotherapy, especially in combination with drugs that target other key immune pathways and are actively planning additional combination cohorts to begin later this year."

"We also recently completed the first stage of the Phase 2 study of CDX-3379 and are pleased that this portion of the study met the clinical criteria that are required to progress the study to the next stage. We look forward to presenting more detailed data from this study at ASCO (Free ASCO Whitepaper) in early June. We are currently conducting a thorough analysis of the overall CDX-3379 program in collaboration with our clinical advisors to determine the optimal path for this candidate. In conclusion, we continue to make considerable progress across our entire pipeline and look forward to updating shareholders over the course of the year," said Marucci.

Recent Highlights:

CDX-1140—a potent CD40 agonist that Celldex believes has the potential to successfully balance systemic doses for good tissue and tumor penetration with an acceptable safety profile.

Enrollment is nearing completion in the monotherapy arm and progressing on track in the CDX-301 combination arm of the Phase 1 dose-escalation study of CDX-1140 with recurrent, locally advanced or metastatic solid tumors and B cell lymphomas. Seven monotherapy dosing cohorts ranging from 0.01 to 1.5 mg/kg have been completed and the dose limiting toxicity (DLT) window successfully cleared; patients are currently being enrolled in the final monotherapy cohort at 3.0 mg/kg. Two combination cohorts in solid tumors (0.09 and 0.18 mg/kg) with CDX-301 have been completed and the DLT window successfully cleared. Patients enrolled in the third cohort at 0.36 mg/kg have been dosed and are currently completing the DLT observation period. Assuming successful clearance, the 0.72 mg/kg combination cohort with CDX-301 should open shortly.

Additional patient enrollment (backfill) has been initiated to characterize the effects of CDX-1140 in the tumor microenvironment and expansion cohorts are being actively planned. Future combination opportunities include PD-1 or PD-L1 inhibitors, chemotherapy, radiation therapy and Celldex’s potent CD27 agonist monoclonal antibody varlilumab.

Data from the ongoing study were presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2019 in April and support that CDX-1140 is a potent activator of CD40 and can be safely administered at doses that Celldex believes will support good tissue and tumor penetration.

CDX-3379—a differentiated human monoclonal antibody designed to block the activity of ErbB3 (HER3). ErbB3 is expressed in many cancers, including head and neck squamous cell cancer (HNSCC) and is believed to be an important receptor regulating cancer cell growth and survival as well as resistance to targeted therapies.

As previously reported, enrollment is complete in the first stage of the Phase 2 study (n=13) of CDX-3379 in advanced HNSCC in combination with Erbitux in Erbitux-resistant patients who have been previously treated with or are ineligible for checkpoint therapy. According to the study’s Simon two-stage design, if at least one patient achieves an objective response in the first stage, enrollment may progress to the second stage. While a confirmed complete response has been documented, Celldex is currently conducting a comprehensive review to inform decisions on potential future development. Celldex plans to present updated data from the study in a poster session at the 2019 American Society for Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting on Saturday, June 1, 2019.

Celldex continues to advance a robust preclinical portfolio with data from multiple programs presented at AACR (Free AACR Whitepaper).

Data from the Company’s CDX-527 bispecific candidate and its TAM program were presented at the AACR (Free AACR Whitepaper) Annual Meeting 2019 in April. CDX-527 uses Celldex’s proprietary highly active anti-PD-L1 and CD27 human antibodies to couple CD27 co-stimulation with blockade of the PD-L1/PD-1 pathway. TAM receptors (Tyro3, Axl, MerTK) are receptor tyrosine kinases (RTKs) expressed in innate immune cells. These receptors have been gaining importance in the immunotherapy field due to their role as checkpoint molecules on macrophages, dendritic cells, and other immune cells, where they can negatively regulate anti-tumor immunity.
First Quarter 2019 Financial Highlights and 2019 Guidance

Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2019 were $85.1 million compared to $94.0 million as of December 31, 2018. The decrease was primarily driven by first quarter cash used in operating activities of $13.2 million, partially offset by $4.2 million in net proceeds from sales of common stock under the Cantor agreement. At March 31, 2019, Celldex had 12.8 million shares outstanding.

Revenues: Total revenue was $1.4 million in the first quarter of 2019 compared to $4.1 million for the comparable period in 2018. The decrease in revenue was primarily due to lower revenue from the contract manufacturing and research and development agreement with the International AIDS Vaccine Initiative and the collaboration agreement with Bristol-Myers Squibb Company.

R&D Expenses: Research and development (R&D) expenses were $11.2 million in the first quarter of 2019 compared to $21.9 million for the comparable period in 2018. The decrease in R&D expenses was primarily due to lower clinical trial, personnel and contract manufacturing costs.

G&A Expenses: General and administrative (G&A) expenses were $4.9 million in the first quarter of 2019 compared to $5.6 million for the comparable period in 2018. The decrease in G&A expenses was primarily due to lower personnel and commercial planning costs.

Intangible Asset and Goodwill Impairments: During the quarter ended March 31, 2018, the Company recorded $18.7 million in non-cash impairment charges related to fully impaired glembatumumab vedotin-related intangible assets and $91.0 million in goodwill impairment charges as the carrying value of the Company’s net assets exceeded the Company’s fair value by an amount in excess of the goodwill asset.

Changes in Fair Value Remeasurement of Contingent Consideration: During the quarter ended March 31, 2019, the Company recorded a $1.5 million loss on fair value remeasurement of contingent consideration primarily due to changes in discount rates and the passage of time. During the quarter ended March 31, 2018, the Company recorded a $13.6 million gain on the fair value remeasurement of contingent consideration primarily due to updated assumptions for glembatumumab vedotin-related milestones as a result of the METRIC study failure and discontinuation of the glembatumumab vedotin program.

Net Loss: Net loss was $17.2 million, or ($1.40) per share, for the first quarter of 2019 compared to a net loss of $118.1 million, or ($12.61) per share, for the comparable period in 2018.

Financial Guidance: Celldex believes that the cash, cash equivalents and marketable securities at March 31, 2019, combined with the anticipated proceeds from future sales of common stock under the Cantor agreement, are sufficient to meet estimated working capital requirements and fund planned operations through 2020. This could be impacted if Celldex elects to pay Kolltan contingent milestones, if any, in cash.

Erbitux is a registered trademark of Eli Lilly & Co.

BioXcel Therapeutics Reports First Quarter 2019 Results and Provides Business Update

On May 7, 2019 BioXcel Therapeutics, Inc. ("BTI" or "Company") (Nasdaq: BTAI), reported its quarterly results for the first quarter ended March 31, 2019 and provided an update on key strategic and operational initiatives (Press release, BioXcel Therapeutics, MAY 7, 2019, View Source [SID1234535818]). BTI is a clinical-stage biopharmaceutical development company utilizing novel artificial intelligence approaches to identify the next wave of medicines across neuroscience and immuno-oncology.

First Quarter 2019 and Recent Highlights:

(BXCL501)- Neuroscience Program-

·Completed dosing of multiple cohorts in Phase 1 pharmacokinetic (bioavailability) and safety study of BXCL501; data announcement expected in coming weeks

·Finalized formulation development and transitioned to automated process for manufacturing of BXCL501 thin film formulation for pivotal study

· Announced proof-of-concept data demonstrating high response rates from three independent Phase 1 studies of intravenously administered dexmedetomidine (Dex) for acute treatment of agitation in patients with schizophrenia, Alzheimer’s disease / dementia and opioid withdrawal symptoms

(BXCL701)- Immuno-Oncology Program-

·Filed investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) on proposed clinical trial of BXCL701, NKTR-214 and avelumab triple combination for treatment of pancreatic cancer along with our partners Pfizer and Merck KGaA, Darmstadt, Germany, and Nektar Therapeutics

·Filed a clinical trial application (CTA) with U.K. health authorities to advance global development of BXCL701 and pembrolizumab (Keytruda) in neuroendocrine prostate cancer (tNEPC)

· Multiple sites opened for tNEPC clinical trial and two sites selected for BXCL701 proof of mechanism study for previously opened INDs

·Presented positive preclinical data on combination of BXCL701 with OX40 agonist at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, showcasing synergistic anti-tumor activity and survival benefit in tumor models

Dr. Vimal Mehta, Chief Executive Officer of BTI said, "During the first quarter of 2019 a number of positive initiatives continued to drive the ongoing clinical advancement of our lead programs, BXCL501 and BXL701."

"We anticipate reporting data from our first-in-human pharmacokinetic (bioavailability) and safety study of BXCL501 in the coming weeks. The dosing of multiple cohorts has been successfully completed. We believe that these results will help lay the groundwork for launching future registration studies as well as supporting BTI’s first New Drug Application (NDA) expected to be filed with the FDA in 2020. Additionally, in order to support the Phase 2/3 registration studies planned to begin in second half of 2019, the process development for BXCL501 sublingual thin film has now been transitioned to an automated robotic aided platform. We intend to employ an automated and scalable manufacturing process for clinical and commercial supply. BXCL501 has demonstrated promising potential with intravenously administered Dex in Phase 1 trials for the acute treatment of agitation in patients with schizophrenia, Alzheimer’s disease/ dementia and opioid withdrawal symptoms, where new effective therapies are desperately needed. We continue to believe that BXCL501 has the potential to be applied to a wide number of neuropsychiatric disorders and look forward to exploring opportunities as we advance development in our initial target indications.

We are pleased to report that we have filed an IND with the FDA for the proposed clinical trial of BXCL701, NKTR-214 and avelumab triple combination for treatment of pancreatic cancer with our partners Pfizer and Merck KGaA and Nektar. The triple combination therapy can potentially be effective by activating both the innate and adaptive immunity in pancreatic cancer patients. We are diligently working with our partners to progress the development of this triple combination therapy. Additionally, our ongoing Phase 1b/2 US study of BXCL701 and pembrolizumab in tNEPC is open for enrollment in multiple clinical trial sites. With the goal of expanding the clinical development of this combination globally, we have filed a CTA with U.K. health authorities. Additionally, we have selected two sites for BXCL701 proof of mechanism study in pancreatic cancer patients.

In March of this year, we presented positive findings at the AACR (Free AACR Whitepaper) Annual Meeting from a preclinical study evaluating the combination of BXCL701 with an OX40 agonist. The findings demonstrated that the combination significantly elevated anti-cancer activity in tumor models compared to the control. These results also provide important validation on BXCL701’s ability to stimulate both innate and adaptive immunity and we expect to further investigate the applicability of this combination."

Dr. Mehta concluded, "We continued to strengthen our management team with the appointment of industry veteran, Dr. Pascal Borderies, as the Vice President, Commercial Development and Medical Affairs who is responsible for designing and executing BTI’s global commercialization and medical affairs strategy, including sales and marketing efforts. We remain intently focused on progressing our assets through clinical development throughout the remainder of the year. We believe our sound strategy, highly skilled leadership team and solid pipeline candidates make us well-positioned for continued growth in 2019 and beyond."

First Quarter 2019 Financial Results

BTI reported a net loss of $7.2 million for the first quarter of 2019, compared to a net loss of $4.3 million for the same period in 2018.

Research and development expenses were $5.6 million for the first quarter of 2019, as compared to $2.9 million for the same period in 2018. The increase was primarily due to a ramp-up of research and development costs, along with increased personnel expenses associated with BTI’s two main drug candidates.

General and administrative expenses were $1.7 million for the first quarter of 2019, as compared to $1.3 million for the same period in 2018. The increase was primarily due to additional payroll and payroll-related expenses and costs associated with operating as a public company.

These results include approximately $682,000 in non-cash share -based compensation charges.

As of March 31, 2019, cash and cash equivalents totaled $36.3 million.

Conference Call:

BTI will host a conference call and webcast today at 8:30 a.m. ET. To access the call please dial (888) 394-8218 (domestic) and (323) 794-2588 (international) and provide the passcode 9482739. A live webcast of the call will be available on the Investors sections of the BTI website at www.bioxceltherapeutics.com. The archived webcast will be available through June 7, 2019.

Upcoming investor conferences:

· UBS Global Healthcare Conference – May 20-22, 2019, New York City

· BXCL501 Investor Day – May 22, 2019, New York City

· BMO Capital Markets Prescription for Success Healthcare Conference – May 25, 2019, New York City

· Jefferies Global Healthcare Conference – June 4-7, 2019, New York City

About BXCL501:

BXCL501 is a first in class, sublingual film of dexmedetomidine, a selective alpha 2a receptor agonist for the treatment of acute agitation. BTI believes that BXCL501 directly targets a causal agitation mechanism and using IV (intravenous) Dex has demonstrated anti-agitation effects in

both preclinical and clinical studies. There is precedent for FDA approval and reimbursement of a non-invasive therapy for the acute treatment of agitation in patients with schizophrenia and bipolar disease, evidenced by regulatory approval of Adasuve, an inhaled version of the antipsychotic loxapine.

About BXCL701:

BXCL701 is a first in class oral immunotherapy with dual mechanisms of action, with an established safety profile from 700 healthy subjects and cancer patients. Designed to stimulate both the innate and acquired immune systems, BXCL701 works by inhibiting dipeptidyl peptidase (DPP) 8/9 and blocking immune evasion by targeting fibroblast activation protein (FAP). Preclinical combination data evaluating BXCL701, a checkpoint inhibitor and other IO agents has demonstrated encouraging anti-tumor activity in multiple tumor types and formation of functional immunological memory. It is under development for tNEPC and pancreatic cancer.

Bellicum Pharmaceuticals Reports First Quarter 2019 Financial Results and Provides Operational Update

On May 7, 2019 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported financial results for the first quarter 2019 and provided an operational update (Press release, Bellicum Pharmaceuticals, MAY 7, 2019, View Source [SID1234535817]).

"We made strong progress in advancing our programs in the first quarter," said Rick Fair, President and Chief Executive Officer of Bellicum Pharmaceuticals. "In our GoCAR-T pipeline, we received acceptance of our abstract with new data from the BPX-601 Phase 1/2 clinical trial for the upcoming ASCO (Free ASCO Whitepaper) Annual Meeting, and made progress toward IND clearance and Phase 1 study start for BPX-603 later this year. In addition, we remain on track to announce topline results from the rivo-cel pediatric registrational trial by the end of the second quarter."

PROGRAM HIGHLIGHTS AND CURRENT UPDATES

BPX-601 GoCAR-T

Bellicum presented initial clinical data from the dose-escalation phase of the Phase 1/2 study of BPX-601 at the Gastrointestinal Cancers Symposium in January that showed a promising safety profile. Additionally, in several patients reported, iMC activation from the administration of rimiducid led to enhanced cell expansion, prolonged cell persistence, and early evidence of clinical activity and disease control. The trial protocol was amended to incorporate a standard lymphodepletion conditioning regimen consisting of cyclophosphamide/fludarabine (Cy/Flu). Updated data from this study—including patients from this Cy/Flu cohort—have been accepted for presentation at ASCO (Free ASCO Whitepaper). As a next step in the study, Bellicum plans to enroll an additional cohort to evaluate repeat rimiducid dosing to re-activate iMC over time, which is intended to deepen and extend the treatment effect. Initial results from this cohort are expected in late 2019.
Controllable Dual-Switch GoCAR-T Product Candidates

Bellicum believes that its next-generation dual-switch GoCAR-T technology may enhance efficacy relative to current generation CAR-T therapy through iMC activation while enabling clinicians to manage certain treatment-emergent toxicities with CaspaCIDe. The company expects to complete an IND application and initiate a Phase 1 clinical trial for BPX-603, a dual-switch GoCAR-T targeting HER2-expressing solid tumors, later this year. The company also expects to submit an IND application by the end of the year for BPX-802, a dual-switch GoCAR-T product candidate targeting an antigen expressed in hematological malignancies.
Rivo-cel

The company expects to report topline results from the pediatric BP-004 study in the second quarter of 2019 and plans to submit Marketing Authorisation Applications (MAAs) for rivo-cel and rimiducid by year-end. Patient recruitment is ongoing in THRIVE, a pivotal randomized global Phase 2/3 clinical trial of rivo-cel in adult and adolescent patients 12 years and older with intermediate and high-risk acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).
First Quarter 2019 Financial Results

Cash Position and Guidance: Bellicum reported cash, restricted cash and investments totaling $78.1 million as of March 31, 2019, compared to $98.0 million at December 31, 2018. Based on current operating plans, Bellicum expects that current cash resources will be sufficient to meet operating requirements through the end of 2019. During the first quarter, Bellicum utilized its at the market financing facility selling 1.4 million shares for net cash proceeds of $4.6 million.

R&D Expenses: Research and development (R&D) expenses were $16.8 million for the first quarter of 2019, compared to $16.5 million for the first quarter of 2018. The higher expenses in the first quarter of 2019 resulted primarily from higher expenditures related to the GoCAR-T platform including initiation of additional clinical sites and costs related to IND filing.

G&A Expenses: General and administrative (G&A) expenses were $7.5 million for the first quarter of 2019 compared to $5.7 million during the comparable period in 2018. The higher expenses in the first quarter 2019 relative to the comparable period in 2018 were primarily due to increased personnel related costs due to hiring additional employees as well as increased costs related to commercialization preparation activities.

Net Loss: Bellicum reported a net loss of $24.5 million for the first quarter of 2019 compared to a net loss of $22.8 million for the first quarter of 2018. The results included non-cash, share-based compensation charges of $2.1 million and $3.6 million for the first quarter of 2019 and 2018, respectively.

Shares Outstanding: At April 30, 2019, Bellicum had 46,009,066 shares of common stock outstanding.

About BPX-601

BPX-601, the company’s first GoCAR-T product candidate, incorporates iMC, Bellicum’s inducible co-activation domain. iMC (inducible MyD88/CD40) is designed to provide a powerful boost to T cell proliferation and persistence and enable the CAR-T to override key immune inhibitory mechanisms, including PD-1 and TGF-beta. BPX-601 is being evaluated as a treatment for solid tumors expressing prostate stem cell antigen (PSCA), including pancreatic, gastric, and prostate cancers.

About Rivo-cel (BPX-501)

Rivo-cel (rivogenlecleucel) is an allogeneic polyclonal T-cell product designed to accelerate immune recovery after HSCT and to reduce relapse of leukemia following a stem cell transplant. The cell treatment contains a diverse repertoire of T cells which may contribute to a robust graft vs. leukemia effect. Rivo-cel’s anti-infective benefits may also reduce morbidity and mortality, as patients are highly susceptible to infection following a transplant. The product’s CaspaCIDe safety switch enables this approach by allowing physicians to reduce the number of alloreactive cells in the event of uncontrolled GvHD. Rivo-cel addresses a major unmet need in adult and pediatric leukemia, lymphoma and inherited blood disease patients following a haploidentical stem cell transplant.

Aptose Reports Results for the First Quarter Ended March 31, 2019

On May 7, 2019 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage company developing highly differentiated therapeutics that target the underlying mechanisms of cancer, reported financial results for the three months ended March 31, 2019 and reported on corporate developments (Press release, Aptose Biosciences, MAY 7, 2019, View Source [SID1234535816]).

The net loss for the quarter ended March 31, 2019 was $5.5 million ($0.14 per share) compared with $6.8 million ($0.23 per share) for the quarter ended March 31, 2018. Total cash and cash equivalents and investments as of March 31, 2019 were $17.0 million. Based on current operations, cash on hand and available sources of capital provide the Company with sufficient resources to fund research and development and operations into 1H 2020.

"With the FDA allowance of our IND for CG-806, our oral, first-in-class pan-FLT3/pan-BTK inhibitor, we entered the second quarter advancing two separate clinical programs with two well-differentiated, small molecule targeted agents for the treatment of patients with hematologic malignancies," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer. "I’m most pleased to announce that we have eleven clinical sites committed to our Phase 1 clinical trial with CG-806 and that we expect to dose our first patient soon. In preclinical studies, CG-806 demonstrated the ability to potently inhibit all wild type and mutant forms of BTK and FLT3 driver kinases and to suppress additional oncogenic signaling pathways upon which cancer cells rely for survival, yet with a precision that avoids targets typically associated with toxicity. In those studies, CG-806 has repeatedly shown superiority to other approved and development stage BTK inhbitors and FLT3 inhibitors. In addition,CG-806 demonstrated safety and tumor elimination in animal models of cancer. Our second compound, APTO-253, is the only clinical-stage molecule shown to directly inhibit expression of the MYC oncogene in a patient with acute myeloid leukemia (AML), without the myelosuppression common to many MYC targeting approaches. Both CG-806 and APTO-253 are investigational products that address unmet needs and substantial market opportunities in hematology."

Key Corporate Highlights

Phase 1 a/b CG-806 Clinical Trial – In March 2019, Aptose announced regulatory allowance from the U.S. Food and Drug Administration (FDA) to initiate a Phase 1 a/b clinical trial program with CG-806, an oral, first-in-class small molecule inhibitor of all known forms of FLT3 and BTK kinases. CG-806 is being developed for the treatment of patients with select hematologic malignancies, including CLL/SLL and non-Hodgkin’s lymphomas (NHL), as well as for patients with relapsed/refractory AML and MDS. The IND allows for immediate testing of CG-806 in patients with certain relapsed or refractory B-cell malignancies, including CLL/SLL and NHL having wild type or C481S mutated forms of the BTK kinase. The first patient will receive 150 mg of oral CG-806 every 12 hours for 28 days. The second patient is planned to receive 300 mg every 12 hours for 28 days, and we anticipate six dose levels during the dose escalation phase of the study. Following identification of the recommended phase 2 dose, we plan to perform four separate expansion trials with up to 25 patients each from four different groups of B-cell malignancy patients. All of these patients will be assessed for safety, tolerance, PK, and a host of biomarkers and scans. Such analyses will characterize the safety and efficacy of CG-806 in this population of patients with B-cell malignancies. In addition, these analyses will inform the dose related plasma exposure levels of CG-806. Once a dose level achieves plasma concentrations that we believe will be therapeutic for AML patients, we plan to present the findings to the FDA and seek to initiate a separate Phase 1 trial for patients with AML and MDS. This strategy avoids treating the acutely ill AML patients to potentially non-efficacious doses and increases the likelihood that responses could be achieved rapidly in the AML/MDS patient population.

Phase 1b Clinical Study of APTO-253 | Favorable Tolerability and Inhibition of MYC Gene Expression in First Patient at Lowest Dose Level | Dosing Begins in Second Patient – Aptose previously announced that dosing had begun in the APTO-253 clinical trial in patients with relapsed or refractory hematologic malignancies. APTO-253 is the only known clinical-stage molecule that can directly inhibit expression of the MYC oncogene, shown to reprogram survival signaling pathways and contribute to drug resistance in many malignancies, including acute AML. The first patient in the trial tolerated the lowest dose of 20 mg/m2 of APTO-253 favorably. In addition, a reduction in MYC gene expression was observed, as well as the induction of p21, an indication of cell cycle arrest and apoptosis, and consistent with target engagement. Dosing of an MDS patient at the second dose level (40 mg/m2 APTO-253) has been initiated, and only one patient will be required at this dose level if no safety issues are observed. APTO-253 is being administered once weekly, over a 28-day cycle, and the study is expected to enroll up to 20 patients with relapsed or refractory AML and high-risk MDS patients. The study is designed to then transition to single-agent expansion cohorts in AML and MDS, followed by combination studies.

New Preclinical Data at AACR (Free AACR Whitepaper) – New preclinical data on CG-806 and APTO-253 were presented in separate poster presentations at the 2019 AACR (Free AACR Whitepaper) Annual Meeting in April. In studies that were conducted in collaboration with the Beat AML Initiative, CG-806 demonstrated significant potency across sub-groups of AML cells, and superior potency when compared to other FLT3 inhibitors, including midostaurin, sorafenib, sunitinib, dovitinib, quizartinib, crenolanib and gilteritinib. Sensitivity of patient cells with IDH1 R132 mutations was an unexpected finding. Additionally, in 28-day GLP toxicity and toxicokinetic studies, CG-806 continued to demonstrate a favorable safety profile. The poster highlights results of combination studies with CG-806 and venetoclax, which demonstrated enhanced killing of primary cancer cells from patients with AML and B-cell cancers. Researchers also presented in vitro studies of APTO-253 focused on its mechanism of action that involves targeting the MYC oncogene. Both AACR (Free AACR Whitepaper) posters are available on the Aptose website.

New $20MM Common Share Purchase Agreement with Aspire Capital Fund, LLC Replaces Prior Agreement – Aptose entered into a new Common Share Purchase Agreement (the "Agreement") with Aspire Capital Fund, LLC ("Aspire Capital") where Aspire Capital has committed to purchase up to $20MM of common shares of Aptose, at Aptose’s request from time to time, for up to 30 months. This Agreement replaces the agreement that Aptose entered into with Aspire Capital on May 30, 2018, which has been terminated by the parties. The Agreement is subject to approval by the Toronto Stock Exchange ("TSX") and NASDAQ, limits the amount of Aptose’s common shares that Aspire can own at one time to 9.99% of the issued and outstanding common shares of the Company, and limits the maximum number of common shares that can be issued under the Agreement to 19.99% of the Company’s outstanding common shares on the date of the Agreement unless shareholder approval is obtained or the shares issued to date once the 19.99% threshold is reached have an average purchase price equal to or exceeding $2.10.

Under the Agreement, no common shares will be sold on the TSX or on other trading markets in Canada. For the purpose of TSX approval, the Company intends to rely on the exemption set forth in Section 602.1 of the TSX Company Manual, which provides that the TSX will not apply its standards to certain transactions involving eligible interlisted issuers on a recognized exchange, such as NASDAQ, provided that the transaction is being completed in compliance with the requirements of such other recognized exchange.

Upon receipt of the TSX and NASDAQ approval, as consideration for Aspire Capital’s obligation under the Agreement, Aptose will issue 171,428 common shares to Aspire Capital as a commitment fee.

Under the terms of the Agreement:
Aptose will control the timing and amount of the sale of common shares to Aspire Capital.
On any business day, Aptose shall have the right to direct Aspire Capital to purchase up to 200,000 common shares with a value not exceeding $500,000. However, upon mutual agreement, Aptose can direct Aspire Capital to purchase up to an additional 2,000,000 common shares.
The purchase price shall be equal to the lesser of: (i) the lowest sale price of the common shares on NASDAQ on the purchase date, or (ii) the average of the three lowest closing sale prices of the common shares on NASDAQ during the 10 business days prior to the purchase date.
In addition to the regular purchases, Aptose shall also have the right to require Aspire Capital to purchase up to an additional 30% of the trading volume of the common shares for the next business day at a purchase price (the "VWAP Purchase Price") equal to the lesser of: (i) the closing price of the common shares on NASDAQ on the VWAP purchase date, or (ii) ninety-seven percent (97%) of the VWAP purchase date’s volume weighted average price on NASDAQ (each such purchase, a "VWAP Purchase").
Aptose shall have the right, in its sole discretion, to determine a maximum number of common shares and set a minimum market price threshold for each VWAP Purchase and there are no limits on the number of VWAP purchases that Aptose may require.
For any business day that the closing sale price of the common shares on NASDAQ is below $0.25, the obligation of Aspire Capital to purchase common shares shall be automatically suspended for that business day only.

Research and development expenses of $3.3 million for the three-month period ended March 31, 2019 were comparable with $3.1 million for the comparative period. Changes to the components of our research and development expenses presented in the table above are primarily as a result of the following events:

In the three-month period ended March 31, 2019, program costs for our CG-806 consisted mostly of costs to complete the preclinical studies and prepare regulatory filings in support of an IND filing, and the manufacturing of drug product for the Phase 1clinical trial. In the comparative period, expenses reflected the completion of two dose range finding studies and the manufacturing of a batch of the drug substance to be used in toxicity studies.
In the three-month period ended March 31, 2019, program costs for our APTO-253 program consisted mostly of costs related to the Phase 1b clinical trial, and manufacturing costs for a second GMP batch of APTO-253. In the comparative period, the Company completed production of a GMP batch of drug product, and initiated necessary studies to present to the FDA in support of removing the clinical hold.
An increase in personnel expenses mostly related to additional clinical research staff to support two Phase 1 clinical trials.
A decrease in stock option compensation related mostly to stock options granted in the three-month period ended March 31, 2018, of which 100,000 with a grant date fair value of $2.03 vested immediately, contributing to higher expenses in that period.

General and administrative expenses of $2.3 million for the three-month period ended March 31, 2019 decreased by approximately $1.4 million compared with $3.7 million for the comparative period, primarily as a result of the following:

General and administrative expenses, excluding non-cash items, decreased by approximately $138.0 thousand, primarily as a result of higher professional and regulatory fees in support of financing activities in the three months ended March 31, 2018, and offset by higher travel, rent and salaries expense in the current period, in support of increased activities in the business.
Stock-based compensation decreased by approximately $1.3 million in the three months ended March 31, 2019, compared with the three months ended March 31, 2018 mostly related to approximately 1,059,000 stock options granted to directors, executive officers and general and administrative employees in the three-month period ended March 31, 2018, of which 750,000 with a grant date fair value of $2.03 vested immediately. In the current period, 1,024,000 stock options were granted to directors, executive officers and general and administrative employees with a grant date fair value of $1.29. Stock options granted by the Company during the three months ended March 31, 2019, vest over four years, except for 335,000 options which vest after one year.
Conference Call and Webcast

Aptose will host a conference call to discuss results for the year and three months ended March 31, 2019 today, Tuesday, May 7, 2019 at 5:00 PM ET. Participants can access the conference call by dialing 1-844- 882-7834 (North American toll free number) and 1-574-990-9707 (International) and using conference ID # 4879249. The conference call can be accessed here and will also be available through a link on the Investor Relations section of Aptose’s website at View Source An archived version of the webcast along with a transcript will be available on the Company’s website for 30 days. An audio replay of the webcast will be available approximately two hours after the conclusion of the call for seven days by dialing 1-855-859-2056, using the conference ID # 4879249.

The press release, the financial statements and the management’s discussion and analysis for the quarter ended March 31, 2019 will be available on SEDAR at www.sedar.com and EDGAR at www.sec.gov/edgar.shtml.

Allogene Therapeutics Reports First Quarter 2019 Financial Results

On May 7, 2019 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) therapies for cancer, reported financial results for the quarter ended March 31, 2019 (Press release, Allogene, MAY 7, 2019, View Source [SID1234535815]).

"We are very pleased with our ability to accelerate the research and development activities for both ALLO-501 and ALLO-715," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "Our focus from day one has been on the acceleration of AlloCAR T therapy to enable an "off-the-shelf" CAR T therapy for patients. In May 2018, we started Allogene with approximately 40 employees. Today, we have over 150 employees, all dedicated to making AlloCAR T therapy a reality."

Recent Highlights

ALLO-501 (anti-CD19 AlloCAR T)

The ALLO-501 Phase 1 ALPHA trial for patients with relapsed/refractory non-Hodgkin lymphoma (NHL) has been initiated. The trial is designed to assess the safety and tolerability at increasing dose levels of ALLO-501 in the most common NHL subtypes of relapsed/refractory large B-cell lymphoma, including diffuse large B-cell lymphoma (DLBCL), and follicular lymphoma (FL). Multiple sites with expertise in CAR T are fully activated and the Company continues to expect to report initial data from the trial in the first half of 2020.

The Company is rapidly progressing the planned second generation of ALLO-501 through preclinical development. The second generation ALLO-501 is devoid of the rituximab off-switch and is expected to be introduced prior to the start of the Phase 2 portion of the ALPHA study.

ALLO-715 (anti-BCMA AlloCAR T)

An Investigational New Drug (IND) application has been submitted to the U.S. Food & Drug Administration (FDA) for ALLO-715, a wholly-owned CAR T product candidate targeting B cell maturation antigen (BCMA) for relapsed/refractory multiple myeloma. The Company remains on track to initiate a Phase 1 trial in 2019.

In April, the Company published in the journal Molecular Therapy preclinical study results validating the potential for an AlloCAR T to treat multiple myeloma. These results demonstrated the ability for ALLO-715 to sustain potent anti-tumor responses in pre-clinical models.

Additional Pipeline Updates

UCART19 (Servier-Sponsored Program in Collaboration with Allogene). Servier is in the process of advancing its supply and re-initiating recruitment for the CALM and PALL trials in relapsed/refractory acute lymphoblastic leukemia. UCART19 is expected to be advanced to potential registrational trials in 2020.

CD70 – At the 2019 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, the Company presented pre-clinical data on its AlloCAR T program targeting CD70, a cancer target that is expressed on both hematologic and solid tumor cells. The data demonstrated the therapeutic potential of an AlloCAR T therapy directed at CD70 in renal cell carcinoma (RCC). The Company plans to select an anti-CD70 AlloCAR T candidate for IND-enabling studies.

Corporate

In May, the Company announced the expansion of its Scientific Advisory Board (SAB) with the appointment of Robert Abraham, Ph.D., Malcolm K. Brenner, M.D., Ph.D., Stephen J. Forman, M.D., and Wendell Lim, Ph.D.

First Quarter Financial Results

Research and development expenses were $23.4 million for the first quarter of 2019, which includes $2.7 million of non-cash stock-based compensation expense.

General and administrative expenses were $13.1 million for the first quarter of 2019, which includes $5.1 million of non-cash stock-based compensation expense.

Net loss for the first quarter of 2019 was $31.6 million, or $0.32 per share, including non-cash stock-based compensation expense of $7.9 million.

As of March 31, 2019, Allogene had $680.7 million in cash, cash equivalents, and investments.

The Company continues to expect full-year 2019 net losses to be between $200 million and $210 million dollars, including estimated non-cash stock-based compensation expense of $45 million to $50 million and excluding any impact from potential business development activities.

Conference Call and Webcast Details

Allogene will host a live conference call and webcast today at 5:30 AM Pacific Time/8:30 AM Eastern Time to discuss financial results and provide a business update. To access the live conference call by telephone, please dial 1 (866) 940-5062 (U.S.) or 1 (409) 216-0618 (International). The conference ID number for the live call is 6179933. The webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.